PL2212424T3 - Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aav - Google Patents
Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aavInfo
- Publication number
- PL2212424T3 PL2212424T3 PL08836776T PL08836776T PL2212424T3 PL 2212424 T3 PL2212424 T3 PL 2212424T3 PL 08836776 T PL08836776 T PL 08836776T PL 08836776 T PL08836776 T PL 08836776T PL 2212424 T3 PL2212424 T3 PL 2212424T3
- Authority
- PL
- Poland
- Prior art keywords
- gene delivery
- motor neurons
- aav vectors
- peripheral injection
- extensive gene
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/04—Centrally acting analgesics, e.g. opioids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/20—Hypnotics; Sedatives
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Genetics & Genomics (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Immunology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Neurology (AREA)
- General Engineering & Computer Science (AREA)
- Neurosurgery (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Pain & Pain Management (AREA)
- Transplantation (AREA)
- Anesthesiology (AREA)
- Hospice & Palliative Care (AREA)
- Rheumatology (AREA)
- Psychiatry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Dermatology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP07301435A EP2058401A1 (de) | 2007-10-05 | 2007-10-05 | Weitverbreitete Genverabreichung an Motorneuronen unter Verwendung einer peripheren Injektion von AAV-Vektoren |
| EP08836776.8A EP2212424B2 (de) | 2007-10-05 | 2008-10-03 | Weitreichende genverabreichung an motorneuronen unter verwendung einer peripheren injektion von aav-vektoren |
| PCT/EP2008/063297 WO2009043936A1 (en) | 2007-10-05 | 2008-10-03 | Widespread gene delivery to motor neurons using peripheral injection of aav vectors |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL2212424T3 true PL2212424T3 (pl) | 2014-11-28 |
Family
ID=39186802
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL08836776T PL2212424T3 (pl) | 2007-10-05 | 2008-10-03 | Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aav |
| PL19158152.9T PL3505635T3 (pl) | 2007-10-05 | 2008-10-03 | Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aav |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL19158152.9T PL3505635T3 (pl) | 2007-10-05 | 2008-10-03 | Rozległe dostarczanie genów do neuronów ruchowych z użyciem wstrzyknięcia obwodowego wektorów aav |
Country Status (19)
| Country | Link |
|---|---|
| US (3) | US9926574B2 (de) |
| EP (5) | EP2058401A1 (de) |
| JP (2) | JP5620819B2 (de) |
| CN (2) | CN101883859A (de) |
| CA (2) | CA2701561C (de) |
| CY (2) | CY1125608T1 (de) |
| DK (2) | DK2212424T3 (de) |
| ES (3) | ES2727034T3 (de) |
| FI (1) | FI2212424T4 (de) |
| FR (2) | FR20C1057I2 (de) |
| HR (2) | HRP20220992T3 (de) |
| HU (2) | HUE059864T2 (de) |
| LT (2) | LT3505635T (de) |
| LU (1) | LUC00285I2 (de) |
| NO (1) | NO2022048I1 (de) |
| PL (2) | PL2212424T3 (de) |
| PT (3) | PT2212424E (de) |
| SI (2) | SI3505635T1 (de) |
| WO (1) | WO2009043936A1 (de) |
Families Citing this family (114)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| EP2019143A1 (de) * | 2007-07-23 | 2009-01-28 | Genethon | Gentherapie des ZNS mit peripher verabreichten AAV-Vektoren |
| EP2058401A1 (de) * | 2007-10-05 | 2009-05-13 | Genethon | Weitverbreitete Genverabreichung an Motorneuronen unter Verwendung einer peripheren Injektion von AAV-Vektoren |
| CA3108119C (en) | 2008-01-15 | 2024-01-16 | The Board Of Trustees Of The Leland Stanford Junior University | Markers of acute myeloid leukemia stem cells |
| PT2242512T (pt) | 2008-01-15 | 2016-07-18 | Univ Leland Stanford Junior | Métodos para manipular a fagocitose mediada por cd47 |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| JP5879256B2 (ja) | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US9272053B2 (en) | 2010-04-23 | 2016-03-01 | University Of Massachusetts | AAV-based treatment of cholesterol-related disorders |
| EP3567106A1 (de) * | 2010-04-23 | 2019-11-13 | University of Massachusetts | Auf das zns abzielende aav-vektoren und verfahren zur verwendung davon |
| CA2833905C (en) | 2010-04-23 | 2019-09-10 | University Of Massachusetts | Multicistronic expression constructs |
| CN107828820B (zh) * | 2010-10-27 | 2022-06-07 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
| US9226976B2 (en) | 2011-04-21 | 2016-01-05 | University Of Massachusetts | RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
| SMT202000445T1 (it) * | 2012-06-19 | 2020-09-10 | Univ Florida | Composizioni e metodi per trattare patologie |
| JP6417322B2 (ja) | 2012-06-21 | 2018-11-07 | アソシアシオン・アンスティテュ・ドゥ・ミオロジーAssociation Institut De Myologie | 遺伝子治療ベクターの広範な遺伝子送達 |
| BR112015002168A2 (pt) | 2012-08-01 | 2017-11-07 | Nationwide Childrens Hospital | liberação intratecal de vírus 9 adeno-associado recombinante |
| US9539307B2 (en) | 2012-09-17 | 2017-01-10 | The Research Institute At Nationwide Children's Hospital | Compositions and methods for treating amyotrophic lateral sclerosis |
| KR20160010526A (ko) * | 2013-05-15 | 2016-01-27 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
| RS63942B1 (sr) | 2013-08-27 | 2023-02-28 | Res Inst Nationwide Childrens Hospital | Proizvodi i postupci za lečenje amiotrofične lateralne skleroze |
| AU2014346987A1 (en) | 2013-11-05 | 2016-06-23 | The Research Institute At Nationwide Children's Hospital | Compositions and methods for inhibiting NF-kB and SOD-1 to treat amyotrophic lateral sclerosis |
| JP6822841B2 (ja) | 2014-03-09 | 2021-01-27 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物 |
| EP2933335A1 (de) * | 2014-04-18 | 2015-10-21 | Genethon | Verfahren zur Behandlung peripherer Neuropathien und Motoneuronerkrankungen |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| US10590420B2 (en) | 2014-07-31 | 2020-03-17 | Association Institut De Myologie | Treatment of amyotrophic lateral sclerosis |
| EP3200830B1 (de) | 2014-10-03 | 2020-09-09 | University of Massachusetts | Hocheffiziente bibliotheksidentifizierte aav-vektoren |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| RU2020140209A (ru) | 2014-10-21 | 2021-01-25 | Юниверсити Оф Массачусетс | Варианты рекомбинантных aav и их применения |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| EP4344741A3 (de) | 2014-11-21 | 2024-08-28 | The University of North Carolina at Chapel Hill | Auf das zentrale nervensystem gerichtete aav-vektoren |
| US10876134B2 (en) | 2014-12-16 | 2020-12-29 | Board Of Regents Of The University Of Nebraska | Gene therapy for juvenile batten disease |
| WO2016131009A1 (en) | 2015-02-13 | 2016-08-18 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| WO2016164642A1 (en) * | 2015-04-08 | 2016-10-13 | The United States Of America, As Represented By The Secretary Of Health And Human Services | Viral gene therapy as treatment for cholesterol storage disease or disorder |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| WO2017058926A1 (en) * | 2015-10-01 | 2017-04-06 | Goleini Inc. | Targeted expression of chloride channels and methods of use thereof |
| DK3365438T3 (da) * | 2015-10-21 | 2021-07-12 | Us Health | Codon-optimeret reduceret-størrelse atp7a cdna og anvendelser ved behandling af kobbertransportlidelser |
| EP3364996B1 (de) | 2015-10-22 | 2021-08-25 | University of Massachusetts | Gegen die prostata gerichtete adeno-assoziierte virusserotypvektoren |
| WO2017070525A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
| EP3368054A4 (de) | 2015-10-28 | 2019-07-03 | Voyager Therapeutics, Inc. | Regulierbare expression unter verwendung des adeno-assoziierten virus (aav) |
| WO2017085317A1 (en) * | 2015-11-19 | 2017-05-26 | Universitat Autonoma De Barcelona | Secreted splicing variant of mammal klotho as a medicament for cognition and behaviour impairments |
| WO2017136536A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| WO2017139643A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| WO2017176929A1 (en) | 2016-04-05 | 2017-10-12 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
| AU2017250298B2 (en) | 2016-04-15 | 2024-10-03 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| WO2017181105A1 (en) | 2016-04-15 | 2017-10-19 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| KR102629418B1 (ko) | 2016-06-13 | 2024-01-25 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 최적화된 cln1 유전자 및 발현 카세트 및 그의 용도 |
| US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| CN110650673B (zh) | 2016-08-30 | 2024-04-09 | 加利福尼亚大学董事会 | 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统 |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| CA3040483A1 (en) | 2016-10-13 | 2018-04-19 | University Of Massachusetts | Aav capsid designs |
| CN108524952A (zh) * | 2017-03-06 | 2018-09-14 | 复旦大学 | 腺相关病毒aav9及其在制备治疗戈谢氏病的制剂中的用途 |
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| EP3793615A2 (de) | 2018-05-16 | 2021-03-24 | Voyager Therapeutics, Inc. | Gerichtete entwicklung von aav zur verbesserung des tropismus für zns |
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